Rare Disease Industry Insights
July 23, 2026 by Dawn Lam
From Fragile Oligonucleotides to an Established Therapeutic Modality Nucleic acid therapeutics (NATs) have evolved from an intriguing scientific concept into a major pharmaceutical modality. More than two decades after the first FDA approval of an oligonucleotide drug, therapeutic oligonucleotides now treat rare diseases, such as spinal muscular atrophy and hereditary transthyretin amyloidosis, to widespread diseases […]
Read more »
Tagged
Oligo Modification By Application
January 6, 2026 by Dawn Lam
In 1977, the laboratories of Richard Roberts and Phillip Sharp independently discovered that the DNA sequences of genes were substantially longer than their respective mRNAs. This led to the discovery of introns and exons, the names coined by Walter Gilbert in 1978 for these ‘intragenic regions’ and ‘expressed regions’ respectively. Gilbert theorized that splicing allowed […]
Get technical content, promotions, and upcoming event news — straight to your inbox.
